Biotechnology company Orchard Therapeutics’ OTL-101 has received a promising innovative medicine designation from the UK Medicines and Healthcare Products Regulatory Agency (MHRA) for the treatment of adenosine deaminase severe combined immunodeficiency.

Commonly known as ADA-SCID or ‘bubble baby’ disease, adenosine deaminase severe combined immunodeficiency is a rare inherited disorder of the immune system.

The disease is caused by mutations in the gene encoding for the adenosine deaminase enzyme, which result in a serious deficiency in white blood cells and lead to life-threatening infections.

OTL-101 is developed by Orchard Therapeutics in collaboration with University College London / Great Ormond Street Hospital (UCL / GOSH) and the University of California, Los Angeles (UCLA).

"With this PIM designation we can potentially make OTL-101 available to UK patients sooner under the EAMS."

The designation indicates that ADA-SCID is a life-threatening condition with high unmet medical need in spite of the currently available treatment options.

Orchard Therapeutics chief scientific officer and UCL / Institute of Child Health Paediatrics professor Bobby Gaspar said: “We are delighted by the designation of OTL-101 as a promising innovative medicine as it recognises the significant unmet need in ADA-SCID, as well as the major potential clinical benefits of OTL-101 over alternative treatment options.”

OTL-101’s designation as a promising innovative medicine is the first step of a two-step process, under which the Orchard treatment can benefit from the early access to medicine scheme (EAMS).

Orchard Therapeutics Business Operations senior vice-president Nicolas Koebel said: “With this PIM designation we can potentially make OTL-101 available to UK patients sooner under the EAMS."